Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial

Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial

<p><strong>Background</strong> Lung delivery of plasmid DNA encoding the CFTR gene complexed with a cationic liposome is a potential treatment option for patients with cystic fibrosis. We aimed to assess the efficacy of non-viral CFTR gene therapy in patients with cystic fibrosis.&...

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Main Authors: Alton, EWFW, Armstrong, DK, Ashby, D, Bayfield, K, Bilton, D, Bloomfield, E, Boyd, A, Brand, J, Buchan, R, Calcedo, R, Carvelli, P, Chan, M, Cheng, S, Collie, D, Cunningham, S, Davidson, H, Davies, G, Davies, J, Davies, L, Dewar, M, Doherty, A, Donovan, J, Dwyer, N, Elgmati, H, Featherstone, R, Gavino, J, Gea-Sorli, S, Geddes, D, Gibson, J, Gill, D, Greening, A, Griesenbach, U, Hansell, D, Harman, K, Higgins, T, Hodges, S, Hyde, S, Hyndman, L, Innes, J, Jacob, J, Jones, S, Keogh, B, Limberis, M, Lloyd-Evans, P, Maclean, A, Manvell, M, McCormick, D, McGovern, M, McLachlan, G, Meng, C, Montero, M, Milligan, H, Moyce, L, Murray, G, Nicholson, A, Osadolor, T, Parra-Leiton, J, Porteous, D, Pringle, I, Punch, E, Pytel, K, Quittner, A, Rivellini, G, Saunders, C, Scheule, R, Sheard, S, Simmonds, N, Smith, K, Smith, S, Soussi, N, Soussi, S, Spearing, E, Stevenson, B, Sumner-Jones, S, Turkkila, M, Ureta, R, Waller, M, Wasowicz, M, Wilson, J, Wolstenholme-Hogg, P
Format: Journal article
Language:English
Published: Elsevier 2015

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